BioMarin Receives Positive CHMP Opinion in Europe to Expand Use of VOXZOGO® (vosoritide) to Treat Children Aged 4 Months and Older with Achondroplasia
- European Commission Approval Decision Expected Q4 2023
- Opinion Based on Positive Results from Global Phase 2 and Ongoing Extension Study
SAN RAFAEL, Calif., [September 15, 2023] – BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia aged 4 months and older whose epiphyses (growth plates) are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the fourth quarter of 2023. VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
“The expansion of VOXZOGO to allow physicians to begin treatment earlier offers the possibility of increased growth and other potential important long-term benefits,” said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. “We look forward to the final approval decision from the European Commission, as well as a decision from the FDA, later this year. These approvals have the potential to further advance care by reaching very young children with achondroplasia.”
Since the introduction of VOXZOGO in 2021, the company has seen strong demand for the medicine worldwide. Over the last few weeks, BioMarin has increased fill-finish commitments and is continuing to work with the company’s fill-finish provider to meet additional demand.
VOXZOGO is the only approved treatment that addresses the underlying cause of achondroplasia. The CHMP based its opinion on data and outcomes from the randomized, double-blind, placebo-controlled Phase 2 clinical trial (111-206) in children aged 4 months to 5 years that showed an improvement in height Z-score of approximately 0.3 standard deviations (SDS) across all age groups after one year of treatment. The application also included longer-term data from the ongoing extension study (111-208).
“For the first time, very young children with achondroplasia may have access to a treatment that has the potential to address the root cause of the condition,” said Klaus Mohnike, professor of pediatrics at Magdeburg University Hospital in Germany and investigator for the VOXZOGO clinical program. “This is a remarkable step forward in our approach to treating achondroplasia.”
Safety profile was favourable for treatment with Vosoritide. The most common adverse events were mild and self-limiting injection site reactions. See the EU (European Union) Summary of Product Characteristics for additional safety information.
Description of Phase 2 Study in Infants and Toddlers
The 52-week phase 2 study enrolled 75 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study consisted of three cohorts by age (24 months to less than 5 years, 6 months to less than 24 months, less than 6 months) and was followed by a subsequent open-label extension trial where all children received active treatment. The objectives of the study were to evaluate safety, tolerability, and the effect of VOXZOGO on growth. The study also evaluated proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries which frequently occur in children with achondroplasia.
About VOXZOGO® (vosoritide) for Injection
In individuals with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). VOXZOGO, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Through BioMarin's broad clinical development program, the company has enrolled 250 children with achondroplasia from eight countries in seven clinical studies evaluating the safety and efficacy of VOXZOGO.
VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
Achondroplasia is the most common form of skeletal dysplasia.1 It is characterized by inhibited endochondral ossification,2 which results in clinical features including disproportionate short stature, shortened limbs and an enlarged head (macrocephaly).2 The condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. 2
Up to 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation.1 The worldwide incidence rate of achondroplasia is about one in 25,000 live births.1 VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.
Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit https://protect-eu.mimecast.com/s/oqvACwkAzIOlq3SVQ8qM?domain=biomarin.eu.
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin’s VOXZOGO program, including (i) BioMarin’s expectations regarding timing and the ability to obtain final approval decision from the European Commission for the marketing authorization to expand the indication for VOXZOGO, and (ii) BioMarin’s expectations regarding the PDUFA Target Action Date with respect to its sNDA for VOXZOGO to expand treatment in the United States to include children with achondroplasia under the age of 5 years; the potential impact of obtaining final approvals for expanded indications from the European Commission and the FDA, including BioMarin’s expectations regarding the number of additional children with achondroplasia that could be prescribed with VOXZOGO and related potential benefits; the potential benefits of VOXZOGO for children with achondroplasia, including the duration of such benefits and potential improvement in proportionality; and the continued clinical development of VOXZOGO. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA, the European Commission and other regulatory authorities; and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
- Cormier-Daire V et al. Optimising the diagnosis and referral of achondroplasia in Europe: European Achondroplasia Forum best practice recommendations. Orphanet J Rare Dis. 2022 Jul 27;17(1):293.
- Savarirayan R et al. C-type natriuretic peptide analogue therapy in children with achondroplasia. N Engl J Med 2019;381(1):25-35.